ALS & Neuromuscular Disorders

Select a condition to begin searching our available clinical trials and research studies.

Amyotrophic lateral sclerosis (ALS)

ALS

Impact of Nuedexta on Bulbar Physiology and Function in ALS

The purpose of this research is to determine if Nuedexta, an FDA approved and routinely prescribed medication, improves speech and swallowing functions in people with ALS.

ALS

Effects of Oral Levosimendan (ODM-109) on Respiratory Function in Patients With ALS

This study will evaluate whether prolonged oral levosimendan can preserve respiratory function more effectively than placebo, resulting in better patient functionality.

ALS

Use of Dalfampridin in Primary Lateral Sclerosis

This study will comprise an 18-week open-label safety and tolerability trial.

ALS

Acute Intermittent Hypoxia and Breathing in Neuromuscular Disease

The investigators are interested in the therapeutic potential of acute intermittent hypoxia (AIH) for individuals with neuromuscular diseases, such as ALS.

ALS

Lung Volume Recruitment Combined With Expiratory Muscle Strength Training in ALS

The purpose of this study is to investigate the effects of two treatment techniques called Expiratory Muscle Strength Training (EMST) and Lung Volume Recruitment (LVR) on breathing, swallowing, speech, and cough function in persons with mild to moderate ALS.

ALS

Delineating Physiologic Mechanisms of Swallowing Impairment and Decline in ALS

The purpose of this study is to evaluate the discriminant ability of several non-invasive screening tools at detecting swallowing impairment in individuals with ALS.

ALS

Natural History of ALS and Other Motor Neuron Disorders

This registry will ask participants diagnosed with ALS for clinical and demographic data to help researchers discover future treatments.

ALS

A Phase 2, Multi-Center, Double-Blind, Randomized, Dose-Ranging, Placebo-Controlled Study to Evaluate the Efficacy, Safety, and Tolerability of CK-2127109 in Patients with Amyotrophic Lateral Sclerosis (ALS)

The purpose of this study is to assess the effect of CK-2127107 versus placebo on respiratory function and other measures of skeletal muscle function in patients with ALS.

ALS

Evaluation of the Safety, Tolerability, Efficacy and Activity of AMX0035, a Fixed Combination of Phenylbutyrate (PB) and Tauroursodeoxycholic Acid (TUDCA), for Treatment of Amyotrophic Lateral Sclerosis (ALS)

AMX0035 is a combination therapy designed to reduce neuronal death through blockade of key cellular death pathways originating in the mitochondria and endoplasmic reticulum (ER). This clinical trial is designed to demonstrate that treatment is safe, tolerable, and able to slow decline in function as measured by the ALSFRS-R. The trial will also assess the effects of AMX0035 on muscle strength, vital capacity, and biomarkers of ALS including markers of neuronal death and neuroinflammation.

Charcot-Marie Tooth

Charcot-Marie Tooth

Study of ACE-083 in Patients with Charcot-Marie-Tooth Disease Types 1 and X

This is a multicenter, phase 2 study to evaluate the safety, tolerability, pharmacodynamics (PD), efficacy, and pharmacokinetics (PK) of ACE-083 in patients with CMT1 and CMTX, to be conducted in two parts. Part 1 is non-randomized, open-label, dose-escalation and Part 2 is randomized, double-blind, and placebo-controlled.

Charcot-Marie Tooth

Assessing Long Term Safety and Tolerability of PXT3003 in Patients With Charcot-Marie-Tooth Disease Type 1A (PLEO-CMT-FU)

This is a multicenter, randomised, placebo-controlled phase III study (CLN-PXT3003-02) to assess the efficacy and safety of PXT3003. The intermediate and high dose of PXT3003 demonstrated an improvement of disability in this patient population in the phase II study.

Muscular dystrophy

Muscular dystrophy

Determination of Accurate Screening Tools for Dysphagia in Oculopharyngeal Muscular Dystrophy

The purpose of this research study is to determine how accurate different tests are at identifying swallowing difficulties in persons with OPMD.

Muscular dystrophy

Establishing Biomarkers and Clinical Endpoints in Myotonic Dystrophy Type 1 (END-DM1)

This study aims to help drug developers successfully design clinical trials and understand how to assess the efficacy of potential therapies. Researchers are trying to learn more about what causes the muscle weakness and stiffness (myotonia) in myotonic dystrophy.

Muscular dystrophy

Studies of skeletal muscle and gastrointestinal dysfunction in myotonic dystrophy and controls

This study is designed to obtain data regarding 2 aspects of the phenotype in myotonic dystrophy (dystrophia myotonica or DM). These are multi-system diseases leading to symptoms in many regions of the body including skeletal muscles, central nervous system and the GI tract.

Muscular dystrophy

Brain imaging and Social cognition in myotonic dystrophy (DM) type 1

This study is designed to assess social cognition deficits in adult onset patients with myotonic dystrophy type 1 and understand possible neuroimaging correlates of such deficits.

MUSCULAR DYSTROPHY

A Phase 2 Randomized, Double-Blind, Placebo-Controlled Study of ACE-083 in Patients with Facioscapulohumeral Muscular Dystrophy

Study A083-02 is a multicenter, Phase 2 study to evaluate the safety, tolerability, pharmacodynamics (PD), efficacy, and pharmacokinetics (PK) of ACE 083 in patients with FSHD to be conducted in two parts. Part 1 is open-label, dose-escalation and Part 2 is randomized, double-blind, and placebo-controlled.

MUSCULAR DYSTROPHY

Phase I/II study to investigate the safety and tolerability, and efficacy of intravenous SGT-001 in male adolescents and children with Duchenne muscular dystrophy

The purpose of this research study is to evaluate a new investigational product called “SGT-001” which is designed to target the underlying cause of DMD in male children and adolescents (ages 4-17), by producing a shortened version of the dystrophin protein.

MUSCULAR DYSTROPHY

Randomised, double-blind, placebo-controlled, multicentre study to evaluate the efficacy and safety of givinostat in ambulant patients with Duchenne Muscular Dystrophy EPIDYS (Epigenetic Rescue of Dystrophin Dysfunction)

The main purpose of this study is to learn how well the investigational drug givinostat works and how safe givinostat is compared to placebo in males 6 years old to 17 years old.

MUSCULAR DYSTROPHY

A Randomized, Double Blind, Placebo-Controlled, Study to Assess the Efficacy, Safety, and Tolerability of RO7239361 in Ambulatory Boys With Duchenne Muscular Dystrophy

We are doing this research study to find out if RO7239361 (previously known as BMS-986089) can help people with Duchenne.